MPNs

A rolling submission of a New Drug Application has been completed for pacritinib which is seeking FDA approval as a treatment for patients with myelofibrosis who have severe thrombocytopenia.

In patients with polycythemia vera, long-term use of the histone deacetylase inhibitor givinostat may be warranted after the agent demonstrated tolerable safety and good efficacy over a 4-year period in an ongoing, multicenter, open-label, single-arm study of patients with a chronic myeloproliferative neoplasm who are positive for a JAK2 V617F mutation.

Naveen Pemmaraju, MD, discusses the results of combining ruxolitinib and navitoclax in different high-risk populations of relapsed/refractory myelofibrosis.

Major thrombosis is a significant danger for patients with MPN who become infected with COVID-19, and antithrombotic prophylaxis does not appear to eliminate the risk.

The FDA has issued a complete response letter to PharmaEssentia Corporation for the company’s biologics license application for ropeginterferon alfa-2b-njft as a potential treatment for patients with polycythemia vera.

Lucia Masarova, MD, discusses the efficacy of CPI-0610 in the phase 2 MANIFEST trial in patients with myelofibrosis.

Ruxolitinib is one of only 2 treatments for MF that significantly improved the survival of patients. Still oncologists see opportunity to prolong survival in these patients even more.

Haris Ali, MD, discusses the results of a study evaluating peri-transplant ruxolitinib in patients with myelofibrosis.

Data from a prospective pilot trial presented at the 2021 Transplantation and Cellular Therapy Meetings show that a maximum dose of 10 mg of ruxolitinib given peri-transplant is a safe and feasible treatment for patients with myelofibrosis.

Raajit K. Rampal, MD, PhD, hematologic oncologist, Memorial Sloan Kettering Cancer Center, reviewed the prognostic tools used to find indicators of response to treatment in patients with myelofibrosis, during a Targeted Oncology Case-Based Peer Perspective Roundtable discussion.

A new meta-analysis confirms that ASXL1 mutations could be an important prognostic indicator in patients with myeloproliferative neoplasms.

JAK inhibitors designed for the treatment of myelofibrosis address the splenic response and constitutional symptoms associated with the disease, but the agents are inherently myelosuppressive and can exacerbate anemia and thrombocytopenia.

In an interview with Targeted Oncology, Naveen Pemmaraju, MD, discussed the potential role of an add-back strategy as treatment of patients with myelofibrosis who no longer benefited from prior ruxolitinib.

Twenty-four or more cycles of fedratinib was well-tolerated in patients with intermediate- or high-risk myelofibrosis treated in either the phase 1/2 TED12015 study or the phase 1 TED12037 study. The long-term safety and tolerability results of fedratinib.

John O. Mascarenhas, MD, discusses the current treatment options for patients with myelofibrosis.

When patients with myelofibrosis who required hematopoietic stem cell transplant received a conditioning regimen of thiotepa, busulfan, and fludarabine along with 2 two alkylating agents, the subsequent transplants were nearly all successful.

Srdan Verstovsek, MD, PhD, discusses how research and future clinical trials can better determine the benefit of ruxolitinib as treatment of patients with myelofibrosis, which is often measured in improvements in the quality of life and spleen reductions.

Patients with myelofibrosis who receive ruxolitinib appear to face a higher risk of second primary malignancy; these risks are suggested to be even higher in men and patients with a history of arterial thrombosis and prolonged hydroxycarbamide exposure.

During a Targeted Oncology Case Based Peer Perspectives event, Andrew Kuykendall, MD, assistant member, Department of Malignant Hematology, H. Lee Moffitt Cancer Center & Research Institute, discussed the use of ruxolitinib as treatment of 67-year-old patients with polycythemia vera.

A study presented during the 2020 ASH Annual Meeting has suggested that certain driver mutations for myeloproliferative neoplasms can be traced back to when they were acquired as early as in utero.

Luspatercept demonstrated clinical efficacy and a tolerable safety profile in patients with myelodysplastic syndrome/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis who were enrolled in the MEDALIST trial.

Naveen Pemmaraju, MD, discusses the next steps for research in the field of myelofibrosis.

Improvements in spleen volume and total symptom score were clinically meaningful with the addition of navitoclax to ruxolitinib in patients with myelofibrosis who no longer benefited from prior ruxolitinib therapy.

During a Targeted Oncology Case Based Peer Perspectives event, Rami Komrokji, MD, explored the use of ruxolitinib (Jakafi) for the treatment of a 68-year-old female patient with myelofibrosis.

Low-dose ruxolitinib appears effective as treatment of patients with myelofibrosis, improving splenomegaly and symptoms with a daily dose ≤ 10 mg.